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Nonviral delivery systems for antisense oligonucleotide therapeutics

BIOMATERIALS RESEARCH (2022)

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Review Chemistry, Multidisciplinary

Chemistry of Lipid Nanoparticles for RNA Delivery Published as part of the Accounts of Chemical Research special issue mRNA Therapeutics

Gaurav Sahay et al.

Summary: This paper discusses the importance of lipid nanoparticles (LNPs) in RNA delivery. LNPs have great potential in mRNA delivery and can be used for the treatment of infectious diseases, cancer, and rare genetic diseases. The paper introduces the core features of LNPs, the theory and methods of LNP self-assembly, the components and overall attributes of LNPs, and provides an outlook on the future of RNA therapies.

ACCOUNTS OF CHEMICAL RESEARCH (2022)

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Review Chemistry, Multidisciplinary

Non-liver mRNA Delivery Published as part of the Accounts of Chemical Research special issue mRNA Therapeutics

James E. L. Dahlman et al.

Summary: This article discusses the potential and challenges of mRNA drugs. mRNA drugs have the ability to prevent or treat various diseases, but their delivery and stability remain problematic. Different drug delivery systems have been developed to overcome these challenges, but improvements are still needed. Currently, mRNA-based COVID-19 vaccines have been successfully developed and approved for clinical use, marking significant progress in the field of mRNA therapeutics.

ACCOUNTS OF CHEMICAL RESEARCH (2022)

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Article Nanoscience & Nanotechnology

Biomineralized Cascade Enzyme-Encapsulated ZIF-8 Nanoparticles Combined with Antisense Oligonucleotides for Drug-Resistant Bacteria Treatment

Yan Zhang et al.

Summary: In this study, GOx&HRP@ZIF-8/ASO nanomaterial was constructed using ZIF-8 material, achieving effective treatment of MRSA infection and filling the gap in the antibacterial application of biological enzymes.

ACS APPLIED MATERIALS & INTERFACES (2022)

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Article Chemistry, Multidisciplinary

Inhaled mRNA Nanoformulation with Biogenic Ribosomal Protein Reverses Established Pulmonary Fibrosis in a Bleomycin-Induced Murine Model

Rui Zhang et al.

Summary: This study reports an inhalable ribosomal protein-based mRNA nanoformulation for the treatment of IPF, which can clear the intrapulmonary ECM and re-epithelialize the disrupted alveolar epithelium to reverse fibrotic foci. The nanoformulation is delivered to fibrotic foci by inhalation and promotes the clearance of collagen and the re-epithelialization of alveoli, leading to improved lung function.

ADVANCED MATERIALS (2022)

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Article Chemistry, Medicinal

The nano delivery systems and applications of mRNA

Mingyuan Li et al.

Summary: The current COVID-19 epidemic has accelerated the application of mRNA technology, highlighting challenges in overcoming mRNA instability in human physiological environments. Research has also shown that mRNA has potential applications beyond COVID-19 vaccines, such as in treating other diseases and genetic disorders.

EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY (2022)

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Article Biotechnology & Applied Microbiology

Regulation of activated microglia and macrophages by systemically administered DNA/RNA heteroduplex oligonucleotides

Rieko Nishi et al.

Summary: In this study, we demonstrate the therapeutic potential of systemically administered Chol-HDO to regulate activated Mg/M9 in neuroinflammation.

MOLECULAR THERAPY (2022)

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Article Pharmacology & Pharmacy

NanoSIMS Imaging Reveals the Impact of Ligand-ASO Conjugate Stability on ASO Subcellular Distribution

Emma Kay et al.

Summary: Conjugating ASOs to GLP1 is an effective method to knock down targets in pancreatic beta-cells. The study found that ASOs separate from GLP1 relatively early in the endocytic pathway, and the stability of the conjugate may affect GLP1-ASO function.

PHARMACEUTICS (2022)

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Article Chemistry, Applied

Co-delivery of doxorubicin and siRNA by all-trans retinoic acid conjugated chitosan-based nanocarriers for multiple synergistic antitumor efficacy

Chang Liu et al.

Summary: In this study, double grafted TMC nanoparticles were developed for the co-delivery of DOX and siRNA, selectively inhibiting the proliferation of tumor cells and exhibiting superior in vivo and in vitro anti-tumor efficiency.

CARBOHYDRATE POLYMERS (2022)

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Review Pharmacology & Pharmacy

Special Section on Pharmacokinetics and ADME of Biological Therapeutics-Minireview Absorption, Distribution, Metabolism, and Excretion of US Food and Drug Administration-Approved Antisense Oligonucleotide Drugs

Julia M. Migliorati et al.

Summary: Absorption, distribution, metabolism, and excretion (ADME) are key biological processes that determine a drug's pharmacokinetic parameters and directly impact its efficacy and adverse reactions. This review focuses on the unique ADME characteristics of antisense oligonucleotide (ASO) drugs, which have diverse modes of drug actions and great potential for treating diseases untreatable by small molecule-based drugs. A meta-analysis of 10 FDA-approved ASO drugs provides comprehensive insights into their formulation, dosage, administration sites, distribution, metabolism, and excretion, improving our understanding of their therapeutic efficacy and potential adverse reactions.

DRUG METABOLISM AND DISPOSITION (2022)

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Article Chemistry, Multidisciplinary

The Unexpected Importance of the Primary Structure of theHydrophobic Part of One-Component Ionizable Amphiphilic JanusDendrimers in Targeted mRNA Delivery Activity

Dapeng Zhang et al.

Summary: Viral and synthetic vectors have significantly impacted genetic nanomedicine, particularly in the rapid development of Covid-19 vaccines. Synthetic vectors have unlimited synthetic capability and exhibit active delivery effects. Studies targeting different organs have shown that by designing the hydrophobic region, IAJDs have increased the activity of gene delivery.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2022)

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Article Infectious Diseases

Cationic lipid-based formulations for encapsulation and delivery of anti-EFG1 2′ OMethylRNA oligomer

Daniela Araujo et al.

Summary: This study aimed to investigate the complexation of cationic liposomes with anti-EFG1 2 ' OMe oligomers and evaluate its efficacy in controlling Candida albicans filamentation in vitro and in vivo using a Galleria mellonella model. The results showed that DOTAP-based lipoplexes were able to release the anti-EFG1 2 ' OMe oligomers and inhibit C. albicans filamentation. In vivo, the DOTAP/DOPC 80/20 rho(chg )= 3 formulation was the most effective, enhancing the G. mellonella survival.

MEDICAL MYCOLOGY (2022)

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Article Medicine, Research & Experimental

Activity and Tissue Distribution of Antisense Oligonucleotide CT102 Encapsulated with Cytidinyl/Cationic Lipid against Hepatocellular Carcinoma

Jing Guan et al.

Summary: This study demonstrated that the Mix/CT102 nanoparticles, encapsulating antisense oligonucleotide CT102, could effectively target tumor cells with high expression of IGF1R. These nanoparticles exhibited efficient inhibition of cell proliferation, downregulation of IGF1R mRNA, and reduction of tumor volume.

MOLECULAR PHARMACEUTICS (2022)

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Review Pharmacology & Pharmacy

Pulmonary delivery of siRNA against acute lung injury/acute respiratory distress syndrome

Makhloufi Zoulikha et al.

Summary: siRNA has potential for treating acute lung injury/acute respiratory distress syndrome by modifying gene expression, but effective delivery remains a challenge.

ACTA PHARMACEUTICA SINICA B (2022)

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Review Chemistry, Applied

Chitosan nanocarriers for microRNA delivery and detection: A preliminary review with emphasis on cancer

Saman Sargazi et al.

Summary: In recent years, gene therapy based on miRNA has emerged as a potential technique for treating co-morbidities. Chitosan, as an effective carrier for miRNA delivery, has been widely utilized. This review discusses the recent progress and future prospects of using chitosan and its derivatives to deliver and detect miRNAs, with a focus on cancer.

CARBOHYDRATE POLYMERS (2022)

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Review Pharmacology & Pharmacy

The role of lipid components in lipid nanoparticles for vaccines and gene therapy

Camilla Hald Albertsen et al.

Summary: The translation discusses the important role of lipid nanoparticles in mRNA vaccines against COVID-19, as well as the contributions of lipid components to the LNP delivery platform. Challenges and strategies related to LNPs in treating various diseases are also explored.

ADVANCED DRUG DELIVERY REVIEWS (2022)

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Article Biochemistry & Molecular Biology

NEAT1 polyA-modulating antisense oligonucleotides reveal opposing functions for both long non-coding RNA isoforms in neuroblastoma

Alina Naveed et al.

Summary: Numerous lncRNAs, including NEAT1, are dysregulated in cancer and are considered therapeutic targets. Researchers have developed ASOs targeting NEAT1_1 to increase NEAT1_2 expression and paraspeckle abundance in neuroblastoma cells, leading to a decrease in cell viability in high-risk neuroblastoma. Furthermore, altering lncRNA polyadenylation events using ASOs shows potential as an anti-cancer strategy.

CELLULAR AND MOLECULAR LIFE SCIENCES (2021)

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Article Nanoscience & Nanotechnology

Harnessing pH-Sensitive Polycation Vehicles for the Efficient siRNA Delivery

Changrong Wang et al.

Summary: The research showed that amphiphilic polycation vehicles with optimal pKa values, strong BCs in the range of pH 5.5-6.5, and low CMCs may be the potential solution for designing efficient vehicles for siRNA delivery. In vitro and in vivo studies supported the effectiveness of EAE5(39/37) in improving siRNA delivery efficiency.

ACS APPLIED MATERIALS & INTERFACES (2021)

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Article Neurosciences

Exosome-mediated delivery of antisense oligonucleotides targeting alpha-synuclein ameliorates the pathology in a mouse model of Parkinson's disease

Jiaolong Yang et al.

Summary: This study demonstrated that exosome-mediated delivery of ASO4 could effectively reduce alpha-synuclein expression, attenuate its aggregation, and improve locomotor functions in a transgenic model of Parkinson's disease.

NEUROBIOLOGY OF DISEASE (2021)

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Article Biochemistry & Molecular Biology

The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration

Paymaan Jafar-nejad et al.

Summary: ASO drugs have been shown to distribute widely in the central nervous system and reduce target RNA in various types of neurons, making them suitable for treating a wide range of neurological diseases.

NUCLEIC ACIDS RESEARCH (2021)

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Article Biochemistry & Molecular Biology

A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping

Yusuke Echigoya et al.

Summary: Duchenne muscular dystrophy (DMD) is a lethal genetic disorder caused by mutations in the DMD gene. Although PMO-ASOs have been approved for clinical use, their applicability remains limited. Establishing a DMD large animal model is crucial for evaluating treatment efficacy.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)

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Article Medicine, Research & Experimental

Single Micelle Vectors based on Lipid/Block Copolymer Compositions as mRNA Formulations for Efficient Cancer Immunogene Therapy

Jingmei Li et al.

Summary: The newly developed DMP micelle delivery system provides a simple and effective approach for efficient mRNA delivery, showing high potential in the treatment of colorectal cancer.

MOLECULAR PHARMACEUTICS (2021)

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Article Biochemistry & Molecular Biology

Perinuclear positioning of endosomes can affect PS-ASO activities

Xue-hai Liang et al.

Summary: Studies have shown that the distribution and activity of PS-ASO in cells are affected by endosome movement and perinuclear positioning. PS-ASO moves linearly along microtubules and covers substantial distances in late endosomes, while perinuclear positioning is necessary for efficient release of PS-ASO.

NUCLEIC ACIDS RESEARCH (2021)

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Article Multidisciplinary Sciences

Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats

Magdalena Derbis et al.

Summary: The study demonstrates that short antisense oligonucleotide steric blockers targeting rCGG(exp) in FXTAS cells can reduce R-loop formation and correct miRNA biogenesis, leading to improved motor behavior and gene expression profile in animal models with minimal toxicity. This suggests a potential therapeutic approach for the treatment of Fragile X-associated tremor/ataxia syndrome.

NATURE COMMUNICATIONS (2021)

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Article Medicine, Research & Experimental

Base-modified aptamers obtained by cell-internalization SELEX facilitate cellular uptake of an antisense oligonucleotide

Keisuke Tanaka et al.

Summary: In this study, aptamers that efficiently internalize into A549 cells were successfully obtained using an evolutionary selection approach. The conjugation of these aptamers with MALAT1-targeting antisense oligonucleotides showed enhanced activity when endosomal/lysosomal escape was improved with the addition of chloroquine. This highlights the importance of endosomal/lysosomal escape for intracellular delivery of antisense oligonucleotides by aptamers.

MOLECULAR THERAPY-NUCLEIC ACIDS (2021)

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Review Chemistry, Multidisciplinary

Development of Polymeric Nanoparticles for Blood-Brain Barrier Transfer-Strategies and Challenges

Weisen Zhang et al.

Summary: Polymeric nanoparticles have shown great potential in overcoming the blood-brain barrier for effective drug delivery, with their unique properties offering high tunability and ease of synthesis. Careful analysis of their performance in transporting drugs across the blood-brain barrier is essential for successful drug delivery to the central nervous system.

ADVANCED SCIENCE (2021)

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Article Multidisciplinary Sciences

Cell membrane coating integrity affects the internalization mechanism of biomimetic nanoparticles

Lizhi Liu et al.

Summary: Cell membrane coated nanoparticles have unique properties that make them attractive for nanomedical applications, but it has been found that most nanoparticles are only partially coated. This partial coating can impact the biological fate of the nanoparticles.

NATURE COMMUNICATIONS (2021)

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Article Chemistry, Multidisciplinary

iRGD-Liposomes Enhance Tumor Delivery and Therapeutic Efficacy of Antisense Oligonucleotide Drugs against Primary Prostate Cancer and Bone Metastasis

Jibin Guan et al.

Summary: The study demonstrates that using iRGD-liposomes as carriers for ASOs in tumor treatment can enhance drug accumulation in tumor tissue and effectively suppress tumor growth without increasing accumulation or toxicity in healthy organs.

ADVANCED FUNCTIONAL MATERIALS (2021)

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Review Chemistry, Multidisciplinary

Functional DNA-Polymer Conjugates

Colette J. Whitfield et al.

Summary: DNA nanotechnology has advanced significantly in the past 30 years, with the combination of solid phase synthesis and the discovery of DNA nanostructures. By bringing together DNA and synthetic polymers, innovative DNA-polymer conjugates have been developed, leading to the creation and application of various advanced nanostructures.

CHEMICAL REVIEWS (2021)

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Review Pharmacology & Pharmacy

Metallic nanoparticles as drug delivery system for the treatment of cancer

Neha Desai et al.

Summary: Metallic nanoparticles show promise in cancer therapy by targeting tumor cells through mechanistic pathways, offering high specificity and efficiency. Recent advancements in nanotechnology highlight their potential for more effective cancer treatment.

EXPERT OPINION ON DRUG DELIVERY (2021)

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Article Biochemistry & Molecular Biology

Therapeutic targeting of STAT3 with small interference RNAs and antisense oligonucleotides embedded exosomes in liver fibrosis

Min Tang et al.

Summary: Hepatic fibrosis is a wound healing response caused by chronic liver injury, where STAT3 plays a crucial role. Using exosomes as a delivery system, this study showed that iExo(siRNA-STAT3) or iExo(mASO-STAT3) effectively targeted STAT3, leading to suppression of liver fibrosis and improved liver function in mice.

FASEB JOURNAL (2021)

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Review Cell Biology

Pre-Clinical and Clinical Applications of Small Interfering RNAs (siRNA) and Co-Delivery Systems for Pancreatic Cancer Therapy

Sepideh Mirzaei et al.

Summary: Pancreatic cancer (PC) is a leading cause of death and the fourth most malignant tumor in men. siRNA can effectively target critical genes in PC progression and enhance the efficacy of chemotherapy and radiotherapy. Loading siRNAs on nanoparticles can protect them from degradation and improve targeted delivery, leading to enhanced therapeutic efficacy in PC treatment.

CELLS (2021)

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Review Biotechnology & Applied Microbiology

Micelles Based on Lysine, Histidine, or Arginine: Designing Structures for Enhanced Drug Delivery

Li Xie et al.

Summary: This review highlights the common approaches to designing functional polymeric micelles based on basic amino acids and their applications as drug carriers for cancer therapy. Different polypeptide architectures are developed for efficient drug loading and delivery, with polylysine- and polyhistidine-based micelles enabling pH-responsive drug release. Additionally, polyarginine can enhance membrane penetration and gas therapy through generating metabolites of nitric oxide (NO).

FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY (2021)

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Review Biotechnology & Applied Microbiology

Antisense technology: an overview and prospectus

Stanley T. Crooke et al.

Summary: Antisense technology is delivering on its promise to treat diseases by targeting RNA, with nine single-stranded ASO drugs approved for commercial use. In addition to rare diseases, ASOs in development are intended to treat common diseases and show potential for increased potency and performance.

NATURE REVIEWS DRUG DISCOVERY (2021)

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Article Chemistry, Multidisciplinary

Delivering Antisense Oligonucleotides across the Blood-Brain Barrier by Tumor Cell-Derived Small Apoptotic Bodies

Yulian Wang et al.

Summary: The study explores the potential of using small apoptotic bodies from brain metastatic cancer cells as carriers for brain-targeted drug delivery, showing high delivery efficiency. By combining anti-TNF-alpha antisense oligonucleotide with sABs, successful enhancement of brain delivery efficiency is achieved. This research provides a new strategy for the treatment of brain diseases.

ADVANCED SCIENCE (2021)

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Article Pharmacology & Pharmacy

Delivery of Oligonucleotides Using a Self-Degradable Lipid-Like Material

Hiroki Tanaka et al.

Summary: This study investigated the optimal lipid composition of LNPs for the delivery of siRNA and ASO, finding that the optimal compositions for siRNA and ASO were different and differed from those reported for mRNA in previous studies. The key processes for successful delivery of mRNA, siRNA, and ASO were identified as extracellular stability, endosomal escape, and cellular uptake, respectively, indicating the importance of optimizing LNP lipid compositions based on the type of nucleic acids being delivered.

PHARMACEUTICS (2021)

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Review Engineering, Biomedical

The evolution of commercial drug delivery technologies

Ava M. Vargason et al.

Summary: This review article discusses how challenges in delivering small molecules, nucleic acids, peptides, proteins, and cells have led to the development of commercial products and are now guiding the delivery of live-cell therapeutics. Drug delivery technologies have enabled the development of pharmaceutical products that enhance therapeutic delivery, minimize off-target effects, and improve patient compliance. Three drug delivery paradigms have been developed to aid in the successful delivery of small molecules and macromolecules, contributing to the initial clinical success of each therapeutic class.

NATURE BIOMEDICAL ENGINEERING (2021)

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Article Chemistry, Multidisciplinary

Spherical Nucleic Acids for Near-Infrared Light-Responsive Self-Delivery of Small-Interfering RNA and Antisense Oligonucleotide

Lei Chen et al.

Summary: This study developed a PSNA for carrier-free and NIR photocontrolled self-delivery of siRNA and ASO, which could selectively release therapeutic agents in tumor cells under NIR light irradiation. PSNA showed promising potential for cancer therapy by combining gene therapy and photodynamic therapy.

ACS NANO (2021)

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Review Pharmacology & Pharmacy

Nucleic acid delivery for therapeutic applications

Akash Gupta et al.

Summary: This review discusses various therapeutic applications of nucleic acids targeted at specific diseases and tissues, highlighting recent technologies that have advanced to late-stage clinical trials and received FDA approval.

ADVANCED DRUG DELIVERY REVIEWS (2021)

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Article Biochemical Research Methods

Gold Nanoparticles as Carriers for Functional RNA Nanostructures

Anna Graczyk et al.

Summary: This study demonstrated for the first time the potential of gold nanoparticle and structural RNA conjugates as tools for gene expression regulation, with the ability to penetrate cells and effectively regulate gene expression. These compounds have the potential to become a new generation of therapeutics with diagnostically useful optical properties and biocompatibility advantages.

BIOCONJUGATE CHEMISTRY (2021)

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Article Engineering, Biomedical

Systemic delivery of mRNA and DNA to the lung using polymer-lipid nanoparticles*

James C. Kaczmarek et al.

Summary: Non-viral vectors can deliver nucleic acids into cells in vivo, with study showing that PBAEs with altered monomer chemistry can effectively deliver DNA or mRNA to lung endothelium in mice. Physical properties like particle size or charge do not explain differences in transfection efficacy, indicating tailored delivery vectors are needed for different nucleic acids. In vivo luciferase expression was significantly higher with mRNA compared to pDNA, highlighting the potential of PBAEs as intracellular delivery materials.

BIOMATERIALS (2021)

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Article Chemistry, Multidisciplinary

The investigation of mRNA vaccines formulated in liposomes administrated in multiple routes against SARS-CoV-2

Hai Huang et al.

Summary: This study developed a liposome-based RBD-mRNA vaccine and examined the impact of different administration routes on immunogenicity. The vaccine successfully induced specific antibodies against SARS-CoV-2 RBD in mice, and the neutralizing capacity against SARS-CoV-2 pseudovirus was efficient. The results showed that administration routes influenced virus neutralization and types of immune responses, suggesting the potential of this vaccine against SARS-CoV-2 with good efficacy and safety.

JOURNAL OF CONTROLLED RELEASE (2021)

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Article Chemistry, Multidisciplinary

Anti-PEG IgM production and accelerated blood clearance phenomenon after the administration of PEGylated exosomes in mice

Sherif E. Emam et al.

Summary: The study demonstrates that injection of PEGylated exosomes induces the production of anti-PEG IgM, leading to decreased blood concentrations of a second dose of PEGylated exosomes and suppressed tumor accumulation. Additionally, the dose of PEGylated exosomes can affect the production of anti-PEG IgM.

JOURNAL OF CONTROLLED RELEASE (2021)

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Article Chemistry, Multidisciplinary

High-throughput evaluation of polymeric nanoparticles for tissue-targeted gene expression using barcoded plasmid DNA

Jayoung Kim et al.

Summary: A new high-throughput method has been developed to evaluate polymeric nanoparticle libraries for tissue-specific gene delivery in vivo. Nanoparticles were found to mainly accumulate in clearance organs, with transfection efficiency not necessarily correlating with tissue accumulation levels. This study validates the effectiveness of the new method in accurately assessing systemic gene delivery.

JOURNAL OF CONTROLLED RELEASE (2021)

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Article Materials Science, Biomaterials

Delivery of antisense oligonucleotides using multi-layer coated gold nanoparticles to methicillin-resistant S. aureus for combinatorial treatment

Marcel Janis Beha et al.

Summary: The study introduces multi-layer coated gold nanoparticles delivering antisense oligonucleotides targeting resistance genes, demonstrating efficient internalization into various types of Gram-positive bacteria with high selectivity and dose-dependence. This approach shows great potential for targeting various MDR bacterial infections through specific gene silencing, as a combinatorial therapeutic with conventional antibiotics.

MATERIALS SCIENCE & ENGINEERING C-MATERIALS FOR BIOLOGICAL APPLICATIONS (2021)

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Article Chemistry, Multidisciplinary

Role of nanotechnology behind the success of mRNA vaccines for COVID-19

Amit Khurana et al.

Summary: The emergency use authorization of two mRNA vaccines by the US-FDA has brought hope in addressing the COVID-19 pandemic. Nanotechnology has played a crucial role in the success of these vaccines, especially with the unique capabilities of lipid nanoparticles. This showcases the potential utility of nanomedicine in tackling global health problems.

NANO TODAY (2021)

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Article Biochemistry & Molecular Biology

Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides

Brooke A. Anderson et al.

Summary: The study found that replacing PS linkages with MsPA in Gapmer ASOs can improve nuclease stability, reduce immune stimulation and cytotoxicity, and extend the duration of ASO action in mice.

NUCLEIC ACIDS RESEARCH (2021)

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Article Biochemistry & Molecular Biology

Golgi-58K can re-localize to late endosomes upon cellular uptake of PS-ASOs and facilitates endosomal release of ASOs

Xue-Hai Liang et al.

Summary: The Golgi apparatus plays a crucial role in the activity of PS-ASO drugs by facilitating the release of these compounds from endosomal organelles. Specific Golgi proteins such as Golgi-58K are shown to be involved in the transport and release of PS-ASOs, highlighting a novel function of Golgi proteins in mediating endosomal release of therapeutic oligonucleotides.

NUCLEIC ACIDS RESEARCH (2021)

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Article Medicine, Research & Experimental

Farnesylthiosalicylic acid-derivatized PEI-based nanocomplex for improved tumor vaccination

Yuang Chen et al.

Summary: This study focuses on nanocomplex-assisted delivery of cancer vaccines, with FTS-PEI showing more effective tumor transfection than other delivery methods and significant presence of transfected cells in lymph nodes. It also demonstrates significant tumor growth inhibition through effective gene delivery to tumors and lymph nodes for neoantigen vaccination.

MOLECULAR THERAPY-NUCLEIC ACIDS (2021)

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Review Chemistry, Multidisciplinary

Multi-Functional Liposome: A Powerful Theranostic Nano-Platform Enhancing Photodynamic Therapy

Xiamin Cheng et al.

Summary: This review discusses the development of liposomes as versatile nano-carriers for drug delivery in order to overcome limitations of PDT and enhance therapeutic effects.

ADVANCED SCIENCE (2021)

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Review Pharmacology & Pharmacy

Lipophilic Polyamines as Promising Components of Liposomal Gene Delivery Systems

Pavel A. Puchkov et al.

Summary: Gene therapy requires an effective and safe nucleic acid delivery vehicle, with cationic amphiphiles being the most commonly used compounds in liposomal formulations. Their structure, consisting of hydrophobic and cationic domains as well as spacer groups, influences delivery efficiency as each component contributes to the overall effectiveness.

PHARMACEUTICS (2021)

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Article Nanoscience & Nanotechnology

Polyamide Microsized Particulate Polyplex Carriers for the 2′-OMethylRNA EFG1 Antisense Oligonucleotide

Daniela Araujo et al.

Summary: The study aimed to develop polyplexes based on porous poly(gamma-butyrolactam) (PA4) or poly(epsilon-caprolactam) (PA6) MPs for the cargo and delivery of anti-EFG1 2'-OMethylRNA ASO. Two types of polyplexes were prepared with anti-EFG1 2'-OMethylRNA molecules, either entrapped or immobilized on prefabricated polyamide MPs. The results confirmed that PA4 and PA6 polyplex MPs can serve as feasible carriers for anti-EFG1 2'-OMethylRNA ASO molecules, maintaining their activity against C. albicans cells through entrapment or immobilization strategies.

ACS APPLIED BIO MATERIALS (2021)

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Review Pharmacology & Pharmacy

Evaluation of nanoparticle drug-delivery systems used in preclinical studies

Nagaraja Sreeharsha et al.

Summary: Multifunctional nanoparticles are considered a promising drug delivery system for sustainable drug release, offering advantages such as tunable structure and size, disease targeting ability, and synergistic effects in multiple drug loading and delivery. These nanoparticulate carriers with specific features enable selective drug delivery with lower potential side effects, demonstrating efficiency and therapeutic outcomes in preclinical models. Challenges associated with translating nanoparticle-based drug carrier responses to clinical application are also highlighted.

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Systemic Brain Delivery of Antisense Oligonucleotides across the Blood-Brain Barrier with a Glucose-Coated Polymeric Nanocarrier

Hyun Su Min et al.

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Advancement on modification of chitosan biopolymer and its potential applications

Nabel A. Negm et al.

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Antisense-Mediated Transcript Knockdown Triggers Premature Transcription Termination

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MXD3 antisense oligonucleotide with superparamagnetic iron oxide nanoparticles: A new targeted approach for neuroblastoma

Sakiko Yoshida et al.

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Hybridization-mediated off-target effects of splice-switching antisense oligonucleotides

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Efficient Delivery of Antisense Oligonucleotides Using Bioreducible Lipid Nanoparticles In Vitro and In Vivo

Liu Yang et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2020)

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Article Materials Science, Biomaterials

Double Click-Functionalized siRNA Polyplexes for Gene Silencing in Epidermal Growth Factor Receptor-Positive Tumor Cells

Yanfang Wang et al.

ACS BIOMATERIALS SCIENCE & ENGINEERING (2020)

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Inhibition of post-trabeculectomy fibrosis via topically instilled antisense oligonucleotide complexes co-loaded with fluorouracil

Kuan Jiang et al.

ACTA PHARMACEUTICA SINICA B (2020)

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Article Biochemistry & Molecular Biology

Chitosan Nanocomplexes for the Delivery of ENaC Antisense Oligonucleotides to Airway Epithelial Cells

A. Katharina Kolonko et al.

BIOMOLECULES (2020)

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Advanced drug delivery 2020 and beyond: Perspectives on the future

You Han Bae et al.

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Polymeric vehicles for nucleic acid delivery

Alexandra S. Piotrowski-Daspit et al.

ADVANCED DRUG DELIVERY REVIEWS (2020)

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Review Pharmacology & Pharmacy

Anti-PEG antibodies: Properties, formation, testing and role in adverse immune reactions to PEGylated nano-biopharmaceuticals

Gergely Tibor Kozma et al.

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Article Biochemical Research Methods

An Oligonucleotide Delivery Platform to Enable Assessment of Intracellular Transcripts in Live Cells by Flow Cytometry

Beverly Z. Packard et al.

CYTOMETRY PART A (2020)

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Review Pharmacology & Pharmacy

Current Status of Antisense Oligonucleotide-Based Therapy in Neuromuscular Disorders

Flavien Bizot et al.

DRUGS (2020)

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Review Pharmacology & Pharmacy

Poly(beta-amino ester)s as gene delivery vehicles: challenges and opportunities

Johan Karlsson et al.

EXPERT OPINION ON DRUG DELIVERY (2020)

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Review Biochemistry & Molecular Biology

Natural Metallic Nanoparticles for Application in Nano-Oncology

Edouard Alphandery

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)

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Review Chemistry, Multidisciplinary

DNA Analogues Modified at the Nonlinking Positions of Phosphorus

Pawan Kumar et al.

ACCOUNTS OF CHEMICAL RESEARCH (2020)

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Article Chemistry, Applied

Exploiting ionisable nature of PEtOx-co-PEI to prepare pH sensitive, doxorubicin-loaded micelles

Naile Ozturk et al.

JOURNAL OF MICROENCAPSULATION (2020)

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Article Chemistry, Multidisciplinary

Effects of pH on the Formation of PIC Micelles from PAMAM Dendrimers

Zhaomei Qiu et al.

LANGMUIR (2020)

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Article Biotechnology & Applied Microbiology

Cellular uptake of self-emulsifying drug-delivery systems: polyethylene glycol versus polyglycerol surface

Julian David Friedl et al.

NANOMEDICINE (2020)

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Review Biochemistry & Molecular Biology

mRNA Vaccine Era-Mechanisms, Drug Platform and Clinical Prospection

Shuqin Xu et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)

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Article Chemistry, Multidisciplinary

Delivery of self-amplifying mRNA vaccines by cationic lipid nanoparticles: The impact of cationic lipid selection

Gustavo Lou et al.

JOURNAL OF CONTROLLED RELEASE (2020)

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Article Pharmacology & Pharmacy

Evaluation of bovine milk extracellular vesicles for the delivery of locked nucleic acid antisense oligonucleotides

Philip Grossen et al.

EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS (2020)

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Article Pharmacology & Pharmacy

Enhanced Antisense Oligonucleotide Delivery Using Cationic Liposomes Grafted with Trastuzumab: A Proof-of-Concept Study in Prostate Cancer

Guillaume Sicard et al.

PHARMACEUTICS (2020)

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Review Medicine, Research & Experimental

Therapeutic Antisense Oligonucleotides Are Coming of Age

C. Frank Bennett

ANNUAL REVIEW OF MEDICINE, VOL 70 (2019)

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Article Engineering, Biomedical

Structural optimization and additional targets identification of antisense oligonucleotide G3139 encapsulated in a neutral cytidinyl-lipid combined with a cationic lipid in vitro and in vivo

Yuan Ma et al.

BIOMATERIALS (2019)

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Review Medicine, Research & Experimental

Targeting RNA: A Transformative Therapeutic Strategy

Wei Yin et al.

CTS-CLINICAL AND TRANSLATIONAL SCIENCE (2019)

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Review Biotechnology & Applied Microbiology

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

Piotr S. Kowalski et al.

MOLECULAR THERAPY (2019)

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Article Chemistry, Multidisciplinary

Boron Dipyrromethene Nano-Photosensitizers for Anticancer Phototherapies

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SMALL (2019)

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Review Chemistry, Multidisciplinary

Development of CLAN Nanomedicine for Nucleic Acid Therapeutics

Cong-Fei Xu et al.

SMALL (2019)

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Article Chemistry, Multidisciplinary

Poly(amine-co-ester) nanoparticles for effective Nogo-B knockdown in the liver

Jiajia Cui et al.

JOURNAL OF CONTROLLED RELEASE (2019)

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Article Chemistry, Multidisciplinary

Oligo(serine ester) Charge-Altering Releasable Transporters: Organocatalytic Ring-Opening Polymerization and their Use for in Vitro and in Vivo mRNA Delivery

Nancy L. Benner et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2019)

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Article Biochemistry & Molecular Biology

Differential uptake, kinetics and mechanisms of intracellular trafficking of next-generation antisense oligonucleotides across human cancer cell lines

Emily Linnane et al.

NUCLEIC ACIDS RESEARCH (2019)

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Review Biochemistry & Molecular Biology

Exosomes

D. Michiel Pegtel et al.

ANNUAL REVIEW OF BIOCHEMISTRY, VOL 88 (2019)

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Review Biochemistry & Molecular Biology

Pharmacokinetics and Clinical Pharmacology Considerations of GalNAc3-Conjugated Antisense Oligonucleotides

Yanfeng Wang et al.

EXPERT OPINION ON DRUG METABOLISM & TOXICOLOGY (2019)

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Article Biochemistry & Molecular Biology

Chitosan nanopolymers: An overview of drug delivery against cancer

Rajasree Shanmuganathan et al.

INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES (2019)

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Review Biotechnology & Applied Microbiology

Cationic micelle: A promising nanocarrier for gene delivery with high transfection efficiency

Haili Wang et al.

JOURNAL OF GENE MEDICINE (2019)

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Article Materials Science, Biomaterials

Magnetically responsive hybrid nanoparticles for in vitro siRNA delivery to breast cancer cells

Milene Dalmina et al.

MATERIALS SCIENCE AND ENGINEERING C-MATERIALS FOR BIOLOGICAL APPLICATIONS (2019)

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Article Cell Biology

Evaluation of off-target effects of gapmer antisense oligonucleotides using human cells

Tokuyuki Yoshida et al.

GENES TO CELLS (2019)

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Review Chemistry, Multidisciplinary

MicroRNA delivery through nanoparticles

Sharon Wei Ling Lee et al.

JOURNAL OF CONTROLLED RELEASE (2019)

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Article Nanoscience & Nanotechnology

Antisense Oligonucleotide-Conjugated Nanostructure-Targeting lncRNA MALAT1 Inhibits Cancer Metastasis

Ningqiang Gong et al.

ACS APPLIED MATERIALS & INTERFACES (2019)

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Review Biotechnology & Applied Microbiology

Nanopharmaceuticals and nanomedicines currently on the market: challenges and opportunities

Fatemeh Farjadian et al.

NANOMEDICINE (2019)

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Article Pharmacology & Pharmacy

Delivery of antisense oligonucleotide using polyethylenimine-based lipid nanoparticie modified with cell penetrating peptide

Shuang Yang et al.

DRUG DELIVERY (2019)

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Article Chemistry, Multidisciplinary

On the Formation and Morphology of Lipid Nanoparticles Containing Ionizable Cationic Lipids and siRNA

Jayesh A. Kulkarni et al.

ACS NANO (2018)

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Review Pharmacology & Pharmacy

Recent preclinical and clinical advances in oligonucleotide conjugates

Kevin Craig et al.

EXPERT OPINION ON DRUG DELIVERY (2018)

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Article Biochemistry & Molecular Biology

Dual pH-sensitive supramolecular micelles from star-shaped PDMAEMA based on beta-cyclodextrin for drug release

Zaishuai Zhou et al.

INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES (2018)

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Review Chemistry, Multidisciplinary

Lipid-polymer hybrid nanocarriers for delivering cancer therapeutics

Tushar Date et al.

JOURNAL OF CONTROLLED RELEASE (2018)

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Article Chemistry, Multidisciplinary

RNA nanoparticle distribution and clearance in the eye after subconjunctival injection with and without thermosensitive hydrogels

Zhanquan Shi et al.

JOURNAL OF CONTROLLED RELEASE (2018)

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Article Chemistry, Multidisciplinary

Evolution of sequence-defined highly functionalized nucleic acid polymers

Zhen Chen et al.

NATURE CHEMISTRY (2018)

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Article Chemistry, Multidisciplinary

Semiconducting Polymer Nanocavities: Porogenic Synthesis, Tunable Host-Guest Interactions, and Enhanced Drug/siRNA Delivery

Haobin Chen et al.

SMALL (2018)

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Article Biochemistry & Molecular Biology

Tunability of Biodegradable Poly(amine-co-ester) Polymers for Customized Nucleic Acid Delivery and Other Biomedical Applications

Amy C. Kauffman et al.

BIOMACROMOLECULES (2018)

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Article Medicine, Research & Experimental

T7 Peptide-Conjugated Lipid Nanoparticles for Dual Modulation of Bcl-2 and Akt-1 in Lung and Cervical Carcinomas

Xinwei Cheng et al.

MOLECULAR PHARMACEUTICS (2018)

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Article Medicine, Research & Experimental

PEG-Peptide Inhibition of Scavenger Receptor Uptake of Nanoparticles by the Liver

Rondine J. Allen et al.

MOLECULAR PHARMACEUTICS (2018)

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Article Biotechnology & Applied Microbiology

Antisense suppression of the nonsense mediated decay factor Upf3b as a potential treatment for diseases caused by nonsense mutations

Lulu Huang et al.

GENOME BIOLOGY (2018)

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Review Pharmacology & Pharmacy

Smart micelleplexes as a new therapeutic approach for RNA delivery

Patricia Pereira et al.

EXPERT OPINION ON DRUG DELIVERY (2017)

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Article Engineering, Biomedical

In vitro evaluation of antisense oligonucleotide functionalized core-shell nanoparticles loaded with -tocopherol succinate

Ebru Kilicay et al.

JOURNAL OF BIOMATERIALS SCIENCE-POLYMER EDITION (2017)

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Review Chemistry, Multidisciplinary

Animal models for evaluation of oral delivery of biopharmaceuticals

Stine Harloff-Helleberg et al.

JOURNAL OF CONTROLLED RELEASE (2017)

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Article Chemistry, Multidisciplinary

Self-assembling complexes between binary mixtures of lipids with different linkers and nucleic acids promote universal mRNA, DNA and siRNA delivery

Thibault Colombani et al.

JOURNAL OF CONTROLLED RELEASE (2017)

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Review Biotechnology & Applied Microbiology

FDA-Approved Oligonucleotide Therapies in 2017

Cy A. Stein et al.

MOLECULAR THERAPY (2017)

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Article Biochemistry & Molecular Biology

Antisense oligonucleotides targeting translation inhibitory elements in 5′ UTRs can selectively increase protein levels

Xue-hai Liang et al.

NUCLEIC ACIDS RESEARCH (2017)

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Review Cell Biology

Disassembly of the Dying: Mechanisms and Functions

Georgia K. Atkin-Smith et al.

TRENDS IN CELL BIOLOGY (2017)

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Review Genetics & Heredity

Advances in the delivery of RNA therapeutics: from concept to clinical reality

James C. Kaczmarek et al.

GENOME MEDICINE (2017)

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Review Engineering, Biomedical

Bio-inspired engineering of cell- and virus-like nanoparticles for drug delivery

Alessandro Parodi et al.

BIOMATERIALS (2017)

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Article Oncology

Co-delivery of microRNA-21 antisense oligonucleotides and gemcitabine using nanomedicine for pancreatic cancer therapy

Yaqing Li et al.

CANCER SCIENCE (2017)

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Review Pharmacology & Pharmacy

Safety of antisense oligonucleotide and siRNA-based therapeutics

Xuan Chi et al.

DRUG DISCOVERY TODAY (2017)

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Review Engineering, Biomedical

Effect of surface properties on liposomal siRNA delivery

Yuqiong Xia et al.

BIOMATERIALS (2016)

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Article Cell & Tissue Engineering

Oncogene Knockdown via Active Loading of Small RNAs into Extracellular Vesicles by Sonication

Tek N. Lamichhane et al.

CELLULAR AND MOLECULAR BIOENGINEERING (2016)

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Article Chemistry, Multidisciplinary

Transcript-activated collagen matrix as sustained mRNA delivery system for bone regeneration

Zohreh Sadat Badieyan et al.

JOURNAL OF CONTROLLED RELEASE (2016)

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Article Biotechnology & Applied Microbiology

Integrated Safety Assessment of 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human Volunteers

Stanley T. Crooke et al.

MOLECULAR THERAPY (2016)

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Article Nanoscience & Nanotechnology

Ultrasound-responsive microbubbles for sonography-guided siRNA delivery

Ping Wang et al.

NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE (2016)

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Article Biotechnology & Applied Microbiology

Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames

Xue-hai Liang et al.

NATURE BIOTECHNOLOGY (2016)

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Review Neurosciences

Extracellular vesicles round off communication in the nervous system

Vivian Budnik et al.

NATURE REVIEWS NEUROSCIENCE (2016)

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Review Chemistry, Multidisciplinary

Nanoparticle-Based Medicines: A Review of FDA-Approved Materials and Clinical Trials to Date

Daniel Bobo et al.

PHARMACEUTICAL RESEARCH (2016)

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Article Biochemistry & Molecular Biology

Disposition and Pharmacokinetics of a GalNAc3-Conjugated Antisense Oligonucleotide Targeting Human Lipoprotein (a) in Monkeys

Rosie Z. Yu et al.

NUCLEIC ACID THERAPEUTICS (2016)

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Article Biotechnology & Applied Microbiology

A Polyethylenimine-Containing and Transferrin-Conjugated Lipid Nanoparticle System for Antisense Oligonucleotide Delivery to AML

Yiming Yuan et al.

BIOMED RESEARCH INTERNATIONAL (2016)

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Review Chemistry, Multidisciplinary

Clearance Pathways and Tumor Targeting of Imaging Nanoparticles

Mengxiao Yu et al.

ACS NANO (2015)

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Review Pharmacology & Pharmacy

Development of a virus-mimicking nanocarrier for drug delivery systems: The bio-nanocapsule

Masaharu Somiya et al.

ADVANCED DRUG DELIVERY REVIEWS (2015)

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Article Engineering, Biomedical

Biomimetic DNA nanoballs for oligonucleotide delivery

Mi-Gyeong Kim et al.

BIOMATERIALS (2015)

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Article Medicine, Research & Experimental

CD33-Targeted Lipid Nanoparticles (aCD33LNs) for Therapeutic Delivery of GTI-2040 to Acute Myelogenous Leukemia

Hong Li et al.

MOLECULAR PHARMACEUTICS (2015)

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Article Medicine, Research & Experimental

Exogenous DNA Loading into Extracellular Vesicles via Electroporation is Size-Dependent and Enables Limited Gene Delivery

Tek N. Lamichhane et al.

MOLECULAR PHARMACEUTICS (2015)

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Article Biochemistry & Molecular Biology

SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice

James Palacino et al.

NATURE CHEMICAL BIOLOGY (2015)

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Review Biotechnology & Applied Microbiology

Targeting protein aggregation for the treatment of degenerative diseases

Yvonne S. Eisele et al.

NATURE REVIEWS DRUG DISCOVERY (2015)

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Article Biochemistry & Molecular Biology

A cytoplasmic pathway for gapmer antisense oligonucleotide-mediated gene silencing in mammalian cells

Daniela Castanotto et al.

NUCLEIC ACIDS RESEARCH (2015)

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Article Engineering, Biomedical

Combinatorial Gene Therapy Accelerates Bone Regeneration: Non-Viral Dual Delivery of VEGF and BMP2 in a Collagen-Nanohydroxyapatite Scaffold

Caroline M. Curtin et al.

ADVANCED HEALTHCARE MATERIALS (2015)

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Review Pharmacology & Pharmacy

PLGA: a unique polymer for drug delivery

Deepak N. Kapoor et al.

THERAPEUTIC DELIVERY (2015)

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Article Biophysics

Micelles of enzymatically synthesized PEG-poly(amine-co-ester) block copolymers as pH-responsive nanocarriers for docetaxel delivery

Xiaofang Zhang et al.

COLLOIDS AND SURFACES B-BIOINTERFACES (2014)

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Article Multidisciplinary Sciences

Defining the Factors That Contribute to On-Target Specificity of Antisense Oligonucleotides

Walt F. Lima et al.

PLOS ONE (2014)

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Article Multidisciplinary Sciences

SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

Nikolai A. Naryshkin et al.

SCIENCE (2014)

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Article Biochemical Research Methods

Neutral Polymeric Micelles for RNA Delivery

Brittany B. Lundy et al.

BIOCONJUGATE CHEMISTRY (2013)

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Review Chemistry, Multidisciplinary

The accelerated blood clearance (ABC) phenomenon: Clinical challenge and approaches to manage

Amr S. Abu Lila et al.

JOURNAL OF CONTROLLED RELEASE (2013)

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Review Clinical Neurology

Developing Therapeutic Antibodies for Neurodegenerative Disease

Y. Joy Yu et al.

NEUROTHERAPEUTICS (2013)

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Review Chemistry, Multidisciplinary

Photoinduced RNA Interference

Yuka Matsushita-Ishiodori et al.

ACCOUNTS OF CHEMICAL RESEARCH (2012)

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Review Pharmacology & Pharmacy

Design of multifunctional non-viral gene vectors to overcome physiological barriers: Dilemmas and strategies

Tao Wang et al.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2012)

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Review Oncology

Silencing disease genes in the laboratory and the clinic

Jonathan K. Watts et al.

JOURNAL OF PATHOLOGY (2012)

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Article Chemistry, Physical

Biodegradable poly(amine-co-ester) terpolymers for targeted gene delivery

Jiangbing Zhou et al.

NATURE MATERIALS (2012)

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Article Chemistry, Multidisciplinary

High Transfection Efficiency of Cationic Lipids with Asymmetric Acyl-Cholesteryl Hydrophobic Tails

Widchaya Radchatawedchakoon et al.

CHEMISTRY-A EUROPEAN JOURNAL (2011)

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Review Nanoscience & Nanotechnology

Cationic Polymer Based Nanocarriers for Delivery of Therapeutic Nucleic Acids

Surendra Nimesh et al.

JOURNAL OF BIOMEDICAL NANOTECHNOLOGY (2011)

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Meeting Abstract Chemistry, Multidisciplinary

Reduction-responsive cationic biodegradable micelles based on PDMAEMA-SS-PCL-SS-PDMAEMA triblock copolymers for gene delivery

Caihong Zhu et al.

JOURNAL OF CONTROLLED RELEASE (2011)

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Review Pharmacology & Pharmacy

Polymeric vectors for ocular gene delivery

Viral Tamboli et al.

THERAPEUTIC DELIVERY (2011)

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Article Biotechnology & Applied Microbiology

Topical delivery of interleukin-13 antisense oligonucleotides with cationic elastic liposome for the treatment of atopic dermatitis

Sung Tae Kim et al.

JOURNAL OF GENE MEDICINE (2009)

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Article Biochemistry & Molecular Biology

A highly effective and long-lasting inhibition of miRNAs with PNA-based antisense oligonucleotides

Su Young Oh et al.

MOLECULES AND CELLS (2009)

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Article Ethics

Are animal models predictive for humans?

Niall Shanks et al.

Philosophy Ethics and Humanities in Medicine (2009)

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Article Pharmacology & Pharmacy

Clusterin antisense complexed with chitosan for controlled intratumoral delivery

Christopher M. Springate et al.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2008)

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Article Chemistry, Medicinal

Comparing in vitro and in vivo activity of 2′-O-[2-(methylamino)-2-oxoethyl]- and 2′-O-methoxyethyl-modified antisense oligonucleotides

Thazha P. Prakash et al.

JOURNAL OF MEDICINAL CHEMISTRY (2008)

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Review Oncology

Photodynamic therapy for treatment of solid tumors - Potential and technical challenges

Zheng Huang et al.

TECHNOLOGY IN CANCER RESEARCH & TREATMENT (2008)

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Article Pharmacology & Pharmacy

Nanoparticles in medicine: Therapeutic applications and developments

L. Zhang et al.

CLINICAL PHARMACOLOGY & THERAPEUTICS (2008)

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Article Biochemistry & Molecular Biology

Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals

Eric E. Swayze et al.

NUCLEIC ACIDS RESEARCH (2007)

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Review Chemistry, Multidisciplinary

Lipid-based nanoparticles for nucleic acid delivery

Weijun Li et al.

PHARMACEUTICAL RESEARCH (2007)

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Article Biochemistry & Molecular Biology

Genospheres: self-assembling nucleic acid-lipid nanoparticles suitable for targeted gene delivery

ME Hayes et al.

GENE THERAPY (2006)

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Article Biochemistry & Molecular Biology

Comprehensive thermodynamic analysis of 3 ' double-nucleotide overhangs neighboring Watson-Crick terminal base pairs

Amanda S. O'Toole et al.

NUCLEIC ACIDS RESEARCH (2006)

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Review Pharmacology & Pharmacy

Dendrimer biocompatibility and toxicity

R Duncan et al.

ADVANCED DRUG DELIVERY REVIEWS (2005)

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Article Pharmacology & Pharmacy

Pharmacokinetics and biodistribution of phosphorodiamidate morpholino antisense oligomers

A Amantana et al.

CURRENT OPINION IN PHARMACOLOGY (2005)

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Article Biotechnology & Applied Microbiology

Phosphoramidate oligonucleotides as potent antisense molecules in cells and in vivo

M Faria et al.

NATURE BIOTECHNOLOGY (2001)

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